Phase 2a study reports favorable safety profile and tolerability of luspatercept in pediatric patients withβ-thalassemia, highlighting potential for treatment.
Key Points
To present the safety data from the non-transfusion-dependent dose-confirmation cohort in pediatric patients receiving luspatercept.
Ongoing phase 2a study with staggered design for patients aged 6 to <18 years
Analysis focused on the NTD dose-confirmation cohort of part A
Patients received luspatercept 1.0 mg/kg subcutaneously every 3 weeks
3 patients enrolled with median age 13 years
All patients experienced at least 1 treatment-emergent adverse event
Safety profile consistent with adult patients, no serious adverse events reported